A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

Last updated: February 10, 2025
Sponsor: Beacon Therapeutics
Overall Status: Active - Recruiting

Phase

2/3

Condition

Eye Disorders/infections

Retinitis Pigmentosa

Posterior Uveitis

Treatment

rAAV2tYF-GRK1-hRPGRco

Control

Clinical Study ID

NCT04850118
AGTC-RPGR-002
  • Ages 12-50
  • Male

Study Summary

This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations.

Eligibility Criteria

Inclusion

General Inclusion Criteria:

  1. Provide written informed consent or assent (per local regulation), prior to theconduct of any study-related procedure. Participants who provide assent must have aparent, guardian, or legal representative provide written informed consent.

  2. Be between 12 and 50 years of age (inclusive) at the time of informed consent andassent (as applicable).

  3. Be male (XY chromosome) and have at least one documented pathogenic or likelypathogenic variant in the RPGR gene.

  4. Have a clinical diagnosis of XLRP.

  5. Be able and willing, as assessed by the Investigator, to follow study instructions,complete study assessments, comply with the protocol, and attend study visits forthe duration of the study. Ocular Inclusion Criteria (Study Eye):

  6. Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200)

  7. Have a LLVA ≤64 letters (approximately Snellen 20/50) in the study eye

  8. Be able to perform all tests of visual and retinal function and structure in botheyes based on the participant's reliability, and fixation, in the study eye per theInvestigator's discretion.

  9. Have detectable baseline mean macular sensitivity .

  10. Have a detectable sub-foveal ellipsoid zone (EZ) line as assessed by SD-OCT in thestudy eye and confirmed by the CRC.

  11. If study eye will be at the discretion of the Investigator and/or Surgeon.

Exclusion

General Exclusion Criteria:

  1. Have other known disease-causing mutations documented in the participant's medicalhistory or identified through a retinal dystrophy gene panel, that in the opinion ofthe Investigator would interfere with the potential therapeutic effect of the studyagent or the quality of the assessments.

  2. For participants with herpes simplex virus (HSV):

  3. Have history of oral or genital herpes and unable and/or unwilling to utilizeprophylactic antiviral medication.

  4. Have a history of ocular herpes.

  5. Have active oral or genital herpes or are currently receiving treatment for HSVinfection.

  6. Have known sensitivity or allergy to systemic corticosteroids or otherimmunosuppressive medications.

  7. Have used anti-coagulant agents that may alter coagulation (e.g., warfarin, heparin,apixaban, or high dose docosahexaenoic acid [DHA; fish oil]) within 7 days prior tostudy treatment administration (ibuprofen, aspirin, or similar are acceptable).

  8. Have used systemic corticosteroids or other immunosuppressive medications within 3months prior to screening and/or intend to use during screening. Corticosteroidsused on an as-needed basis administered by insufflation, inhalation or localadministration to the skin and mucosa such as Symbicort (budesonide/formoterol),Flonase (fluticasone propionate), and skin creams and ointments containingcorticosteroids shall not be exclusionary.

  9. If sexually active or planning to become sexually active, are unwilling to usebarrier contraception for 3 months following treatment administration.

  10. Are currently participating or recently participated in any other research

  11. Have previously received any AAV gene therapy product, stem cell therapy, cell-basedtherapy, or similar biologics.

  12. Have significant media opacity impacting evaluation of the retina or vitreous.administration.

  13. Had intraocular surgery within 90 days of study treatment administration.

  14. Have any active ocular/intraocular infection or inflammation (e.g., severeblepharitis, infectious conjunctivitis, keratitis, scleritis, endophthalmitis,idiopathic or autoimmune associated uveitis, or herpetic lesions).

  15. Have a history of corticosteroid-induced raised IOP of >25 mmHg followingcorticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.

  16. Have any artificial retinal implant or prosthesis.

  17. Have absence of clear ocular media and/or inadequate pupil dilation to facilitategood quality SD-OCT images.

  18. Have any history of rhegmatogenous retinal detachment.

  19. Have myopia (spherical equivalent) exceeding -10 diopters (or axial length of >30 mmif the Principal Investigator [PI] deems it appropriate to measure) or presence ofpathologic myopia in the study eye.

  20. Have passed the Low Contrast Ora-VNC mobility course at ≤0.35 lux light level ineither eye or binocularly at any screening visit.

Study Design

Total Participants: 75
Treatment Group(s): 2
Primary Treatment: rAAV2tYF-GRK1-hRPGRco
Phase: 2/3
Study Start date:
March 14, 2024
Estimated Completion Date:
October 31, 2029

Study Description

This study is a randomized, controlled, masked, multi-center study evaluating and comparing 2 doses of AGTC-501 to an untreated control group. A single subretinal injection of AGTC-501 Dose 1 or Dose 2 will be administered in participants in 2 treatment groups while participants in the untreated control group will be followed and evaluated, after which they will be evaluated to determine eligibility to receive treatment with AGTC-501 Dose 2.

Approximately 75 eligible male participants between 12 and 50 years of age (inclusive) will be randomized in a 1:1:1 ratio to 1 of 3 groups.

Connect with a study center

  • Sydney Eye Hospital

    Sydney, New South Wales 2000
    Australia

    Site Not Available

  • Royal Victorian Eye & Ear Hospital

    East Melbourne, Victoria 3002
    Australia

    Site Not Available

  • Hadassah Medical Center

    Jerusalem,
    Israel

    Site Not Available

  • Radboud University Medical Center

    Nijmegen,
    Netherlands

    Site Not Available

  • Rotterdam Ophthalmic Institute

    Rotterdam,
    Netherlands

    Site Not Available

  • Moorfields Eye Hospital

    London, EC1V 2PD
    United Kingdom

    Site Not Available

  • The Retina Clinic London, Institute of Ophthalmology, University College London

    London, W1G7LB
    United Kingdom

    Site Not Available

  • Oxford Eye Hospital

    Oxford, OX39DU
    United Kingdom

    Active - Recruiting

  • Retina Macula Institute of Arizona

    Scottsdale, Arizona 85255
    United States

    Site Not Available

  • University of San Diego

    San Diego, California 92110
    United States

    Site Not Available

  • Colorado Retina Associates

    Denver, Colorado 80230
    United States

    Site Not Available

  • University of Florida

    Gainesville, Florida 32611
    United States

    Site Not Available

  • University of Florida Health Jacksonville, Department of Ophthalmology

    Jacksonville, Florida 32209
    United States

    Active - Recruiting

  • Bascom Palmer Eye Institute- University of Miami

    Miami, Florida 33136
    United States

    Active - Recruiting

  • Midwest Eye Institute (Retina Partners Midwest)

    Carmel, Indiana 46290
    United States

    Site Not Available

  • Wilmer Eye Institute at Johns Hopkins

    Baltimore, Maryland 21287
    United States

    Site Not Available

  • Boston Children' Hospital

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Ophthalmic Consultants of Boston

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Mayo Clinic

    Rochester, Minnesota 55905
    United States

    Active - Recruiting

  • Duke Eye Center

    Durham, North Carolina 27705
    United States

    Site Not Available

  • Cincinnati Eye Institute

    Cincinnati, Ohio 45242
    United States

    Active - Recruiting

  • Cleveland Clinic - Cole Eye Institute

    Cleveland, Ohio 44195
    United States

    Site Not Available

  • Cole Eye Institute - Cleveland Clinic

    Cleveland, Ohio 44195
    United States

    Active - Recruiting

  • Casey Eye Institute, OHSU

    Portland, Oregon 97239
    United States

    Active - Recruiting

  • Casey Eye Institute, Oregon Health and Sciences University

    Portland, Oregon 97239
    United States

    Site Not Available

  • Mid Atlantic Retina

    Philadelphia, Pennsylvania 19107
    United States

    Active - Recruiting

  • University of Pittsburgh

    Pittsburgh, Pennsylvania 15219
    United States

    Active - Recruiting

  • Retina Consultants of Texas

    Bellaire, Texas 77401
    United States

    Active - Recruiting

  • Retina Foundation of the Southwest

    Dallas, Texas 75231
    United States

    Active - Recruiting

  • Baylor Eye Institute

    Houston, Texas 77030
    United States

    Active - Recruiting

  • Retina Consultants of San Antonio Texas

    San Antonio, Texas 78240
    United States

    Active - Recruiting

  • Retina Consultants of Texas- San Antonio

    San Antonio, Texas 78240
    United States

    Active - Recruiting

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